Mattijs Bulcaen, Phéline Kortleven, Ronald B Liu, Giulia Maule, Elise Dreano, Mairead Kelly, Marjolein M Ensinck, Sam Thierie, Maxime Smits, Matteo Ciciani, Aurelie Hatton, Benoit Chevalier, Anabela S Ramalho, Xavier Casadevall I Solvas, Zeger Debyser, François Vermeulen, Rik Gijsbers, Isabelle Sermet-Gaudelus, Anna Cereseto, Marianne S Carlon
Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause cystic fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines...
April 24, 2024: Cell reports medicine